From: Clinical evidence for orphan medicinal products-a cause for concern?
 | Number of indications (%) |
---|---|
Prevalence of the rare disease in which the indication is authorized | |
 Between 4/10 000 and 5/10 000 | 1 (1.3%) |
 Between 3/10 000 and 4/10 000 | 3 (3.8%) |
 Between 2/10 000 and 3/10 000 | 5 (6.4%) |
 Between 1/10 000 and 2/10 000 | 14 (17.9%) |
 Between 1/10 000 and 2/10 000 | 42 (53.8%) |
Requested protocol assistance | |
 Yes | 22 (28.2%) |
 No | 15 (19.2%) |
 Not reported | 41 (52.6%) |
Performed dose finding study | |
 Yes | 44 (56.4%) |
 No | 25 (32.0%) |
 Not reported | 9 (11.5%) |